uniQure

We had a wonderful weekend at #HDCongress25, where we engaged with #huntingtonsdisease clinicians, researchers, and advocates and gained valuable insights as we plan the path forward for our investigational #genetherapy candidate AMT-130.

Today Dr. Victor Sung, one of the investigators in our AMT-130 #genetherapy trial, gave an oral presentation on the AMT-130 topline pivotal data as part of the #HDCongress2025 HD Clinical Trials Update.

We couldn’t be more excited to be a sponsor of, and attend, the Huntington Study Group HD Clinical Research Congress this weekend in Nashville, where we look forward to connecting with the #huntingtonsdisease research community and sharing a scientific presentation about our investigational #genetherapy candidate, AMT-130. #HDCongress2025

Earlier today we announced that the pivotal Phase I/II study of our #genetherapy candidate AMT-130 in #huntingtonsdisease met its primary and a key secondary endpoint in the high-dose, with favorable trends across additional clinical measures. https://lnkd.in/ew2g5-26

Today we announced positive topline data from the pivotal study of our investigational #genetherapy candidate AMT-130 for #huntingtonsdisease, which met its prespecified primary endpoint, with high-dose AMT-130 demonstrating a 75 percent slowing of disease progression as measured by cUHDRS compared to an external control. https://lnkd.in/ew2g5-26

uniQure Exec. Dir of Patient Advocacy Dan Leonard was invited recently to co-host a session at Rare Rounds – a collaboration between Global Genes and the Termeer Institute. The event was part of the Global Genes Rare Drug Development Symposium and brought together patient advocates and biotech leaders to discuss issues of mutual importance, such as Accelerated Approval pathways for rare diseases.

This past weekend, uniQure presented for the first time clinical data on our Fabry disease #genetherapy AMT-191 at the #ICIEM2025 conference in Kyoto, Japan. There we presented preliminary results from our first cohort, showing that all patients discontinued enzyme replacement therapy and that AMT-191 has a manageable safety profile at the highest dose. The team returns home from a long trip invigorated by the support of the #Fabry medical community and looking forward to continuing our work on behalf of Fabry patients. https://lnkd.in/erRJ7vEG

Today we announced initial safety and exploratory efficacy data from the Phase I/IIa trial of our investigational #genetherapy AMT-191 for #Fabry disease. All patients in the first cohort discontinued enzyme replacement therapy and preliminary data show that AMT-191 has a manageable safety profile at the highest dose. https://lnkd.in/erRJ7vEG

At today's International #Epilepsy Congress poster session, uniQure's Dr. Firas Taha presented a case study on the GenTLE Phase I/IIa Clinical Trial of our #genetherapy AMT-260. https://lnkd.in/erBr-wDB #IEC2025

The uniQure team is happy to join the global #epilepsy community at this year's International Epilepsy Congress in Lisbon! uniQure is proud to present updates on AMT-260, our investigational #genetherapy for drug-resistant mesial temporal lobe epilepsy. If you're in attendance, stop by uniQure’s booth in space 219 to learn more. #IEC2025